Rare Disease Research, LLC | Atlanta, GA
Status and phase
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About
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
Enrollment
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Inclusion criteria
Exclusion criteria
Note: Other inclusion and exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
24 participants in 4 patient groups, including a placebo group
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Central trial contact
PepGen
Data sourced from clinicaltrials.gov
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